Status:
COMPLETED
Establishment of Human Cellular Disease Models for Morquio Disease
Lead Sponsor:
CENTOGENE GmbH Rostock
Conditions:
Morquio Disease
Eligibility:
All Genders
12+ years
Brief Summary
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
Detailed Description
The mucopolysaccharidoses are a group of inherited lysosomal storage disorders. Lysosomes function as the primary digestive units within cells. Enzymes within lysosomes break down or digest particular...
Eligibility Criteria
Inclusion
- Informed consent will be obtained from the patient or the parents before any study related procedures
- Patients of both genders older than 12 months
- Patient has a diagnosis of Morquio disease
Exclusion
- No Informed consent from the patient or the parents before any study related procedures
- Patient is younger than 12 months
- Patient has no diagnosis of Morquio disease
Key Trial Info
Start Date :
October 26 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2019
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT03872713
Start Date
October 26 2018
End Date
December 1 2019
Last Update
April 9 2021
Active Locations (1)
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1
Childrens Hospital and Institute of Child Health, Ferozepur Road
Lahore, Pakistan, 54600